WO2025166323A3 - Crispr-related methods and compositions targeting lipoprotein (a) expression - Google Patents

Crispr-related methods and compositions targeting lipoprotein (a) expression

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Publication number
WO2025166323A3
WO2025166323A3 PCT/US2025/014247 US2025014247W WO2025166323A3 WO 2025166323 A3 WO2025166323 A3 WO 2025166323A3 US 2025014247 W US2025014247 W US 2025014247W WO 2025166323 A3 WO2025166323 A3 WO 2025166323A3
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WO
WIPO (PCT)
Prior art keywords
crispr
expression
lipoprotein
related methods
compositions targeting
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
PCT/US2025/014247
Other languages
French (fr)
Other versions
WO2025166323A2 (en
Inventor
Paul Joseph WRIGHTON
Heather Dawn MACLEOD
Judith Anne NEWMARK
Chrysa M. LATRICK
Mark Thomas JONES
Xiaoqi Sun
John Anthony Zuris
Christopher Wilson
Bryant CHICA
Michael Christopher JASKOLKA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Editas Medicine Inc
Original Assignee
Editas Medicine Inc
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Filing date
Publication date
Application filed by Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of WO2025166323A2 publication Critical patent/WO2025166323A2/en
Publication of WO2025166323A3 publication Critical patent/WO2025166323A3/en
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present disclosure relates to CRISPR-related genome editing systems and components for editing and/or modulating the expression of a target nucleic acid sequence of interest, for example, an LPA target nucleic acid sequence encoding the Apolipoprotein(a) protein. The present disclosure is also directed to methods and applications thereof in connection with the treatment and/or management of disease using the described genome editing systems.
PCT/US2025/014247 2024-02-02 2025-02-02 Crispr-related methods and compositions targeting lipoprotein (a) expression Pending WO2025166323A2 (en)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US202463549247P 2024-02-02 2024-02-02
US63/549,247 2024-02-02
US202463557142P 2024-02-23 2024-02-23
US63/557,142 2024-02-23
US202463631436P 2024-04-08 2024-04-08
US63/631,436 2024-04-08
US202463643353P 2024-05-06 2024-05-06
US63/643,353 2024-05-06
US202463718386P 2024-11-08 2024-11-08
US63/718,386 2024-11-08

Publications (2)

Publication Number Publication Date
WO2025166323A2 WO2025166323A2 (en) 2025-08-07
WO2025166323A3 true WO2025166323A3 (en) 2025-09-18

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PCT/US2025/014247 Pending WO2025166323A2 (en) 2024-02-02 2025-02-02 Crispr-related methods and compositions targeting lipoprotein (a) expression

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Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017158422A1 (en) * 2016-03-16 2017-09-21 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
WO2019204668A1 (en) * 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
WO2022132773A1 (en) * 2020-12-14 2022-06-23 New York Genome Center, Inc. Chemically modified crispr-cas13 guide rnas
WO2023180904A1 (en) * 2022-03-21 2023-09-28 Crispr Therapeutics Ag Methods and compositions for treating lipoprotein-related diseases
WO2023220207A2 (en) * 2022-05-10 2023-11-16 Editas Medicine, Inc. Genome editing of cells
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
WO2024197065A2 (en) * 2023-03-20 2024-09-26 Verve Therapeutics, Inc. In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease
WO2024206676A1 (en) * 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa

Family Cites Families (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU747597B2 (en) 1998-07-20 2002-05-16 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
CA2532228C (en) 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
EP1750673B1 (en) 2004-05-17 2009-12-02 Tekmira Pharmaceuticals Corporation Liposomal formulations comprising dihydrosphingomyelin and methods of use thereof
JP4764426B2 (en) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド Cationic lipids and methods of use
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
WO2006074546A1 (en) 2005-01-13 2006-07-20 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
CN101267805A (en) 2005-07-27 2008-09-17 普洛体维生物治疗公司 System and method for making liposomes
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
CN111808084A (en) 2008-11-10 2020-10-23 阿布特斯生物制药公司 Novel lipids and compositions for delivery of therapeutic agents
US8722082B2 (en) 2008-11-10 2014-05-13 Tekmira Pharmaceuticals Corporation Lipids and compositions for the delivery of therapeutics
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
KR101766408B1 (en) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 Improved lipid formulation
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
WO2013012680A1 (en) 2011-07-15 2013-01-24 3M Innovative Properties Company An electrical connector
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
ES2745769T3 (en) 2014-03-10 2020-03-03 Editas Medicine Inc CRISPR / CAS related procedures and compositions for treating Leber 10 congenital amaurosis (LCA10)
HUE060907T2 (en) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
JP6637988B2 (en) 2014-11-18 2020-01-29 アークトゥルス セラピューティクス， インコーポレイテッド Ionizable cationic lipids for RNA delivery
WO2016197133A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles
PT3313829T (en) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Lipids and lipid nanoparticle formulations for delivery of nucleic acids
JP6799058B2 (en) * 2015-09-21 2020-12-09 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Allele selective gene editing and its use
HRP20230209T1 (en) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
US9834510B2 (en) 2015-12-30 2017-12-05 Arcturus Therapeutics, Inc. Aromatic ionizable cationic lipid
WO2017117528A1 (en) 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
LT3436077T (en) * 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipid nanoparticle formulations for crispr/cas components
CA3040337A1 (en) 2016-10-26 2018-05-03 Curevac Ag Lipid nanoparticle mrna vaccines
EP3532103B1 (en) 2016-10-26 2025-12-03 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
WO2018119163A1 (en) 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10227302B2 (en) 2017-02-09 2019-03-12 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018183901A1 (en) 2017-03-30 2018-10-04 The Government Of The United States Of America As Represented By The Secretary Of The Army Nucleic acid vaccine composition comprising a lipid formulation, and method of increasing the potency of nucleic acid vaccines
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
WO2018191719A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipid delivery of therapeutic agents to adipose tissue
CN110799492B (en) 2017-04-28 2023-06-27 爱康泰生治疗公司 Novel carbonyl lipid and lipid nanoparticle formulations for nucleic acid delivery
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
EP4501322A3 (en) 2017-08-17 2025-04-16 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
EP3746554A1 (en) 2018-01-30 2020-12-09 Editas Medicine, Inc. Systems and methods for modulating chromosomal rearrangements
WO2019191780A1 (en) 2018-03-30 2019-10-03 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for manufacturing lipid nanoparticles and liposomes
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
AU2019376006A1 (en) 2018-11-09 2021-06-03 Arbutus Biopharma Corporation Cationic lipids containing silicon
KR20210105889A (en) 2018-11-09 2021-08-27 아뷰터스 바이오파마 코포레이션 Lipid Nanoparticle Formulation
CA3118559A1 (en) 2018-11-09 2020-05-14 Arbutus Biopharma Corporation Lipid nanoparticle formulations
EP3876999A4 (en) 2018-11-09 2022-08-31 Arbutus Biopharma Corporation Negatively charged peg-lipid conjugates
PT3908568T (en) 2019-01-11 2024-09-30 Acuitas Therapeutics Inc Lipids for lipid nanoparticle delivery of active agents
SG11202110036RA (en) 2019-03-19 2021-10-28 Arcturus Therapeutics Inc Method of making lipid-encapsulated rna nanoparticles
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CN114901253A (en) 2019-08-14 2022-08-12 爱康泰生治疗公司 Improved lipid nanoparticles for delivery of nucleic acids
IL293946A (en) * 2019-12-18 2022-08-01 Editas Medicine Inc Transgenic cells for therapy
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2022011156A1 (en) 2020-07-10 2022-01-13 Genevant Sciences Gmbh Lipid nanoparticles for delivering therapeutics to lungs
ES3054438T3 (en) 2020-07-16 2026-02-03 Acuitas Therapeutics Inc Cationic lipids for use in lipid nanoparticles
EP4196128A4 (en) 2020-08-14 2024-06-12 Arcturus Therapeutics, Inc. METHOD FOR LYOPHILIZATION OF LIPID DNANOPARTICLES
IL301253A (en) 2020-09-13 2023-05-01 Arcturus Therapeutics Inc Lipid nanoparticles encapsulation of large rna
AU2021403156A1 (en) 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles
CA3219053A1 (en) 2021-05-05 2022-11-10 Arcturus Therapeutics, Inc. Lipid compositions comprising peptide-lipid conjugates
US20220378702A1 (en) 2021-05-05 2022-12-01 Arcturus Therapeutics, Inc. Peptide-lipid conjugates
KR20240008872A (en) 2021-05-06 2024-01-19 아크투루스 쎄라퓨틱스, 인크. Ionizable Cationic Lipids for RNA Delivery
US20240376499A1 (en) 2021-07-09 2024-11-14 University Of Massachusetts Modular prime editing systems for genome engineering
TW202313967A (en) 2021-07-30 2023-04-01 美商亞克圖羅斯醫療公司 Rna vaccines
WO2023076898A1 (en) 2021-10-25 2023-05-04 The Broad Institute, Inc. Methods and compositions for editing a genome with prime editing and a recombinase
AU2022381750A1 (en) 2021-11-03 2024-05-16 Arcturus Therapeutics, Inc. Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis
IL312507A (en) * 2021-11-03 2024-07-01 Intellia Therapeutics Inc RNAS is a different guide to gene editing
WO2023086514A1 (en) 2021-11-11 2023-05-19 Arcturus Therapeutics, Inc. Ionizable cationic lipids for rna delivery
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US20250059131A1 (en) 2021-12-16 2025-02-20 Acuitas Therapeutics, Inc. Fluorinated cationic lipids for use in lipid nanoparticles
EP4700017A2 (en) 2021-12-16 2026-02-25 Acuitas Therapeutics Inc. Fluorinated cationic lipids for use in lipid nanoparticles
EP4532702A1 (en) 2022-06-02 2025-04-09 University of Massachusetts High fidelity nucleotide polymerase chimeric prime editor systems

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017158422A1 (en) * 2016-03-16 2017-09-21 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
WO2019204668A1 (en) * 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
WO2022132773A1 (en) * 2020-12-14 2022-06-23 New York Genome Center, Inc. Chemically modified crispr-cas13 guide rnas
WO2023180904A1 (en) * 2022-03-21 2023-09-28 Crispr Therapeutics Ag Methods and compositions for treating lipoprotein-related diseases
WO2023220207A2 (en) * 2022-05-10 2023-11-16 Editas Medicine, Inc. Genome editing of cells
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
WO2024197065A2 (en) * 2023-03-20 2024-09-26 Verve Therapeutics, Inc. In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease
WO2024206676A1 (en) * 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
AGEELY EMAN A. ET AL: "Gene editing with CRISPR-Cas12a guides possessing ribose-modified pseudoknot handles", NATURE COMMUNICATIONS, vol. 12, no. 1, 1 January 2021 (2021-01-01), UK, XP093186433, ISSN: 2041-1723, Retrieved from the Internet <URL:https://www.nature.com/articles/s41467-021-26989-z> DOI: 10.1038/s41467-021-26989-z *
DOERFLER ALEXANDRIA M. ET AL: "LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study; with supplemental information", MOLECULAR THERAPY- METHODS & CLINICAL DEVELOPMENT, vol. 27, 13 October 2022 (2022-10-13), GB, pages 337 - 351, XP093282655, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2022.10.009 *

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